Amgen has received the US Food and Drug Administration (FDA) approval for Uplizna to treat adults with Immunoglobulin G4-related disease (IgG4-RD).
Uplizna is a humanised monoclonal antibody (mAb) that causes targeted and sustained depletion of key cells that contribute to the underlying disease process.
IgG4-RD is a chronic immune-mediated inflammatory condition that can affect multiple organs and may lead to fibrosis and permanent damage.
It is the second approved indication for Uplizna, which was previously approved for treating AQP4-IgG+ Neuromyelitis Optica Spectrum Disorder (NMOSD) in June 2020.
In addition, the drug also received FDA Orphan Drug Designation for generalised myasthenia gravis (gMG), with regulatory submissions anticipated latter half of this year.
Amgen research and development executive vice president Jay Bradner said: “The FDA approval of Uplizna marks a significant turning point for IgG4-RD patients and physicians who now have a proven treatment that targets a key driver of the disease, reducing the risk of flares and reliance on harmful long-term steroid use.
“We are proud to deliver a therapy that has the potential to significantly improve care for patients with IgG4-RD and remain encouraged by Uplizna’s broader potential in other immune-mediated diseases, including neuromyelitis optica spectrum disorder and generalised myasthenia gravis.
“This approval underscores Amgen’s ongoing commitment and leadership in developing innovative treatments targeting CD19+ B-cells across multiple therapeutic areas.”
The FDA approval of Uplizna follows FDA Breakthrough Therapy Designation, based on high unmet medical need.
It is backed by data from the MITIGATE trial, a multicentre study that evaluated the efficacy and safety of Uplizna in reducing flare risks in adults with IgG4-RD.
The clinical trial validated Uplizna’s potential to decrease disease activity by reducing flares while maintaining its safety profile.
The study also includes an optional three-year open-label treatment period and a safety follow-up period of up to two years post-discontinuation.
The study principal investigator John Stone said: “Targeting CD19+ B cells with UPLIZNA has proven to be a highly effective approach to help address the pathophysiology of IgG4-RD.
“The clinical community now has an FDA-approved therapeutic innovation for patients that targets underlying disease mechanisms and helps to control disease activity by reducing flares in IgG4-RD.
“Now, our work begins in raising awareness of this disease so that patients can access the right treatment as early as possible, avoiding a long and often harmful diagnostic journey.”
